olipudase alfa

Olipudase alfa is a recombinant human acid sphingomyelinase and the first and only enzyme replacement therapy for the treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann–Pick disease. ASMD is a rare lysosomal storage disorder caused by mutations in the SMPD1 gene, resulting in deficient activity of acid sphingomyelinase and accumulation of sphingomyelin in various organs such as the liver, lungs, spleen, kidneys, and bone marrow. Olipudase alfa works by catalyzing the hydrolysis of accumulated sphingomyelin into ceramide and phosphocholine, thereby reducing pathological substrate buildup in affected tissues. It does not cross the blood-brain barrier and thus does not treat central nervous system symptoms. The drug has demonstrated efficacy in improving visceral organ function (e.g. reduced spleen/liver volume; improved lung function) in both adult and pediatric patients with ASMD[2][6][7][8].

Brand names

Xenpozyme

Other names

recombinant human acid sphingomyelinaserhASM